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Gene editing inside the body for alpha-1 antitrypsin deficiency.

1 report so far. New developments are added to this thread as they happen.

  1. Nov 3, 2026
    ONE-TIME EDIT PARTLY FIXES THE GENE IN A PATIENTEarly trial · 1 patient

    One-time gene-editing drip partly fixed an inherited liver gene in a man

    Doctors in Germany report that a single infusion edited the faulty gene behind alpha-1 antitrypsin deficiency in a 66-year-old man. About half the gene copies checked in his liver were corrected.